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US 7,115,258 B2Granted
Vesicular monoamine transporter gene therapy in parkinson's disease
AssigneeArch Development Corporation
InventorUn Jung Kang
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Abstract
The present invention provides methods and compositions for the therapeutic intervention of Parkinson's disease. More particularly, methods of making and sequestering dopamine are disclosed. Additionally, methods of genetically modifying donor cells by gene transfer for grafting into the central nervous system to treat defective, diseased or damaged cells are disclosed. Methods and compositions for carrying out such gene transfer and grafting are described.
Claims6
A method for increasing dopamine levels in a mammal, said method comprising:
- (a) obtaining cells from the mammal;
- (b) transforming said cells with a first polynucleotide encoding L-amino acid decarboxylase (AADC) and a second polynucleotide encoding vesicular monoamine transporter (VMAT) under conditions suitable for the expression of AADC and VMAT, wherein said polynucleotides each are under the transcriptional control of a promoter;
- and
- (c) implanting said transformed cells into said mammal;
- whereby AADC converts L-DOPA in vivo to dopamine and VMAT sequesters said dopamine in endosomes of said cells, which sequestered dopamine releases over a longer duration of time than from cells without storage of L-DOPA.
2 more elements
A method of treating Parkinson's disease in a human subject comprising:
- (a) obtaining cells from said subject;
- (b) transforming said cells with a first polynucleotide encoding L-amino acid decarboxylase (AADC) and a second polynucleotide encoding vesicular monoamine transporter (VMAT) under conditions suitable for the expression of AADC and VMAT, wherein said polynucleotides are under the transcriptional control of a promoter;
- (c) implanting said transformed cells into said subject;
- and
- (d) providing L-DOPA to said subject.
2 more elements
Dependencies are inferred from the claim text.